Raptor Pharmaceuticals Corp. RPTP presented efficacy results at 36 months from the phase II/III CYST-HD study on RP103 for the treatment of patients with Huntington's disease. The study is being conducted in collaboration with Centre Hospitalier Universitaire d'Angers, France.
Data from the study showed that the candidate was generally well tolerated in patients. Moreover, the Total Motor Score (TMS) was consistent with the improvement in functional measures, which included a 23% reduction in the rate of decline in Total Functional Capacity (TFC) and a 46% reduction in the rate of deterioration on the Independence Scale. While the Independence Scale revealed a statistically significant improvement, the TMS score was statistically insignificant.
Based on this 36-month data, the company intends to seek regulatory guidance on the clinical development and regulatory pathway of RP103 in both the U.S. and the EU.
RP103 has Orphan drug designation in both the U.S. and the EU for Huntington's disease.
We note that Raptor is also evaluating RP103 in a phase II study for the treatment of patients with mitochondrial diseases, including Leigh syndrome. Second interim results from the study are expected in the first quarter of 2016.
According to information provided by the company, about 30,000 patients and 100,000 patients are estimated to be affected by Huntington's disease in the U.S. and in the world, respectively.
Raptor currently carries a Zacks Rank #3 (Hold). Some better-ranked stocks in the health care sector include Achillion Pharmaceuticals, Inc. ACHN, Anika Therapeutics Inc. ANIK and Corcept Therapeutics Incorporated CORT, each sporting a Zacks Rank #1 (Strong Buy).
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